Genervon Successfully Completes its Phase 2a Clinical Trial for ALS

AAN 2014

April 28, 2014 02:35 AM Eastern Daylight Time

PASADENA, Calif--(BUSINESS WIRE)--Genervon Biopharmaceuticals LLC (“Genervon”) today announced that it has
successfully completed its Phase 2a clinical trial for amyotrophic
lateral sclerosis (“ALS”) disease modification. A full analysis of the
trial’s results is expected to be completed in the third quarter of
2014, but preliminary data suggests that Genervon’s novel, proprietary,
multi-target biological drug candidate, GM604, shows significant promise
for treating ALS.

The randomized, double-blinded trial was conducted at two sites with six
ALS patients participating at each site. Each patient received six
intravenous doses of either GM604 (eight patients) or placebo (four
patients) over two weeks and was then evaluated at three time points
over the following ten weeks. Disease progression was measured by ALS
Functional Rating Scale –Revised (ALSFRS-R), Time Up and Go (TUG), and
Forced Vital Capacity (FVC).

According to the preliminary data, ten weeks following completion of
dosing without further treatment, clinical measurements of ALS disease
progression remained the same and unchanged from the baseline in two of
the trial’s eight patients treated with GM604, while the rates of
degradation of those clinical measurements had slowed in five of the
remaining six treated patients.

Additional information regarding the trial is available on request, and
a final analysis of the preliminary data will be presented later this
year.

In the interim Genervon is planning the next trial for GM604. GM 604 has
already received orphan drug and fast track designation from FDA for ALS
indication. Genervon intends to partner with one or more pharmaceutical
companies in connection with this effort.

Discussions with prospective partner candidates are expected to occur at
the American Academy of Neurology Convention in Philadelphia from April
26th through May 3rd; interested partner firms are encouraged to contact
Genervon to schedule a discussion.